Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions – Fifth Meeting

In response to a Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine is conducting a study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union.

This is the fifth meeting of the committee of experts and will be conducted virtually. The meeting is a closed session and will not include a public session.

The committee will evaluate:

• Flexibilities, authorities, or mechanisms available to regulators in the United States and the European Union applicable to rare diseases or conditions;
• The consideration and use of supplemental data submitted during review processes in the United States and the European Union, including data associated with open label extension studies and expanded access programs specific to rare diseases or conditions;
• An assessment of collaborative efforts between United States and European Union regulators related to: product development programs under review; policies under development and those recently issued; and scientific information related to product development or regulation.

Based on its information gathering and internal deliberations, the committee will develop a report with its findings, conclusions, and recommendations for actions that Congress, federal agencies, the pharmaceutical industry, and nongovernmental organizations can take to support collaborative efforts.

Please visit the National Academies’ Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union event page for more information.